Within five years, likely well before we start treating patients with their own genetically corrected stem cells created from induced pluripotent stem cells, I am expecting to see new drugs discovered using iPS cells enter the clinical pipeline.

It’s only been two years since three labs (ours included) created the first human iPS cells through genetic reprogramming of skin cells. Since our creation of the first repository of disease-specific iPS cells in 2008, the number of diseases being modeled in iPS cells has increased to dozens worldwide.

By creating iPS cells from patients with specific diseases, researchers can model that disease in a culture dish and observe its earliest beginnings. In 2008, the laboratory of George Q. Daley, MD, PhD, Director of Stem Cell Transplantation Program, reported creating a collection of iPS cell lines from patients with 10 different diseases. The lines are under active study at Children’s Hospital Boston, and are available to scientists around the world, housed at a core facility at the Harvard Stem Cell Institute. The Daley Lab has also taught scores of scientists how to make the cells themselves.

The production of induced pluripotent stem cells is often imprecise, yielding many incompletely reprogrammed cells. Now, Thorsten Schlaeger, PhD, and George Daley, MD, PhD, of Children’s Stem Cell Program have developed a battery of tests to ensure that he has the real thing: pure pluripotent stem cells. The new work creates a standard of analysis in the field. Read more.